Science Spotlight: Ranking AAV capsids for liver gene therapy

“Near-clinical” human liver models have revealed differences in transduction efficiency across liver cell types and disease states, among five AAV capsids commonly used in liver gene therapies.

The models, published in Nature Biotechnology, comprised whole human livers, derived from healthy and patient donors, that were unable to be used for transplantation. The authors perfused the organs with oxygen-packed red blood cells to maintain physiological conditions. The liver remained viable for about four days under the conditions...